Oral ONC201 in Relapsed/Refractory Multiple Myeloma


ONC201 is an oral, first-in-class small molecule with demonstrated antitumor activity in preclinical models of difficult-to-treat solid and liquid tumors. ONC201 acts on dopamine receptors in the body to trigger a variety of known anti-cancer pathways without imparting significant toxicity. This is a Phase 1/2 open-label study of ONC201 administered orally once every week incombination with dexamethasone to patients with relapsed/refractory multiple myeloma.

  • In Phase I ONC201 is intended to be administered as oral capsules once every week at doses of 375 mg or 625 mg.
  • Dexamethasone will be administered same day of each ONC201 administration. 
  • Once a recommended phase 2 dose (RP2D) has been determined in Phase I based on a favorable benefit/risk ratio (anticipated to be 625mg ONC201 with 20mg dexamethasone), the study will progress to Phase 2 of the study.
SparkCures ID 828
Trial Phase Phase 1/2
Enrollment 42 Patients
Trial Sponsors
  • Oncoceutics, Inc.
NCT Identifier


Am I Eligible?

The following criteria is a partial list of reasons why patients may or may not be eligible to participate in this clinical trial. Further evaluation with a medical professional will be required to determine full eligibility.

The following criteria is provided for health care professionals.

Inclusion Criteria:

  1. Confirmed evidence of disease progression from immediately prior multiple myeloma therapy or refractory to the immediately prior treatment.
  2. Measurable disease M protein component in serum (at least 0.5 g/dL) and/or urine (if present) (>=0.2 g excreted in a 24 hour collection sample).
  3. All previous therapies for cancer, including radiotherapy, major surgery and investigational therapies discontinued for ≥ 14 days (≥ 28 days for mitomycin C or nitrosoureas) before study entry, and all acute effects of any prior therapy resolved to baseline severity or Grade ≤ 1 Common Terminology Criteria for Adverse Events (CTCAE v4.03), except alopecia or parameters defined in this eligibility list.
  4. Age ≥ 18 years.
  5. ECOG performance status ≤ 1.
  6. Adequate organ and marrow function as defined below:
    1. Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1)
    2. Platelets ≥75,000/mm3 without platelet transfusion ≤ 3 days prior to C1D1
    3. Hemoglobin >8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1
    4. Total serum bilirubin <1.5 X upper limit of normal (ULN)
    5. AST (SGOT)/ALT (SGPT) ≤2 X ULN; ≤ 5 X ULN if there is liver involvement secondary to tumor
    6. Serum creatinine ≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
    7. Serum or urine pregnancy test (for females of childbearing potential) negative ≤7 days of starting treatment
  7. Ability to understand and the willingness to sign a written informed consent document and comply with the study scheduled visits, treatment plans, laboratory tests and other procedures.
  8. Female patients must be surgically sterile or be postmenopausal, or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. The decision of effective contraception will be based on the judgment of the principal investigator or a designated associate.

Exclusion Criteria:

  1. Active inflammatory gastrointestinal disease, chronic diarrhea (unless related to underlying malignancy or prior related treatment) or history of abdominal fistula, gastrointestinal perforation, peptic ulcer disease, or intra-abdominal abscess within 6 months prior to study enrollment. Gastroesophageal reflux disease under treatment with proton pump inhibitors is allowed.
  2. Pregnancy or breast feeding.
  3. Current active treatment in another clinical study.
  4. Active bacterial, fungal or viral infection including hepatitis B (HBV), hepatitis C (HCV)
  5. Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness.
  6. Active or prior plasma cell leukemia (defined as either 20% of peripheral WBC comprised of plasma/CD138+ cells or an absolute count of 2 x 10^9/L).
  7. Solitary bone or solitary extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia.
  8. Subjects with serum calcium (corrected for albumin) ≥ 12 mg/dL
  9. Any of the following in the previous 6 months: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack or symptomatic pulmonary embolism.
  10. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results, or in the judgment of the investigator would make the patient inappropriate for entry into the study.

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